A Decade of Impact: Patient Worthy’s 10-Year Journey Empowering the Rare Disease Community
Launched in May 2015, Patient Worthy set out with a bold mission: to amplify the voices of rare disease and chronic illness patients, and to
JumpStart
JumpStart
The Orphan Disease Center’s JumpStart program serves to establish and progress research agendas in emerging and neglected rare diseases. The JumpStart program partners with patient groups and families to address gaps by identifying key opinion leaders and introducing new researchers to a disease. The program can help facilitate the development of animal models, establish a patient alliance or a patient registry, and organize symposia. Due to limited resources, ODC is unable to provide financial support. JumpStart primarily offers scientific advisement and network outreach.
Condition Awareness & Advocacy
Here is a list of conditions this partner raises awareness and advocacy for:
- Emerging and neglected rare diseases
Resources & Support
Patient Worthy Posts on Rare Disease
Vitamin B3 Shows Promise in Slowing Rapid Aging in Rare Disease Patients
A recent study has revealed that vitamin B3 could offer hope for patients suffering from a rare genetic disorder that causes accelerated aging. As reported
Eddie Vedder Shines Light on Rare Disease Advocacy in “Matter of Time” Documentary
Pearl Jam front-man Eddie Vedder takes center stage in the new documentary “Matter of Time,” offering fans and viewers a rare, intimate glimpse into his
FDA Unveils Plans to Modernize Rare Disease Regulations
The FDA is taking significant steps to update and streamline its regulatory approach to rare diseases, according to a recent article from BioCentury.com. Recognizing the
Dancing Through Diagnosis: Alnylam’s Givlaari Campaign Puts Rare Disease in the Spotlight
Alnylam Pharmaceuticals has launched a unique promotional campaign for Givlaari, its treatment for acute hepatic porphyria (AHP), that swaps the usual clinical narratives for something
Global Health Milestone: United Nations Adopts Landmark Resolution on Rare Diseases
In a significant stride for global health, the United Nations recently adopted a historic resolution dedicated to rare diseases. This move, as reported by Medics
